A mother's plea to the FDA for quicker action in order to save her sons

Austin and Max Leclaire are brothers and both have the rare childhood disease, Duchenne, a type Muscular Dystrophy causing the muscles to weaken over time ultimately leading to the inability to walk, heart failure, and death. Duchenne affects one out of every 3,500 boys born in the U.S. and has a 100 percent fatality rate.

For the first time, there is a treatment for Duchenne in clinical trials called eteplirsen. 12 boys have been receiving this treatment and Max is one of them. Along with the other boys, the drug is visibly helping Max with his condition making him stronger. However, his brother, Austin, did not qualify to be one of the patients in the first phase of testing and is progressively getting worse.

Currently, the FDA will not grant a New Drug Application for eteplirsen even though it's effects are proven successful and there is a high demand. "The FDA has the power to grant conditional approval for a drug if the medicine is 'reasonably expected' to improve or extend lives" says Tracy Seckler, mother of another son with Duchenne, Charley, whom is also fighting to get the drug cleared so her own son can begin treatment.

There is currently an accelerated approval plan to get eteplirsen approved by the FDA and readily available for other Duchanne patients in need, like Austin.